Pharmaceutical Research

, Volume 22, Issue 4, pp 499–511

Method Validation and Measurement of Biomarkers in Nonclinical and Clinical Samples in Drug Development: A Conference Report

  • Jean W. Lee
  • Russ S. Weiner
  • Jeff M. Sailstad
  • Ronald R. Bowsher
  • Dean W. Knuth
  • Peter J. O’Brien
  • Jean L. Fourcroy
  • Rakesh Dixit
  • Lini Pandite
  • Robert G. Pietrusko
  • Holly D. Soares
  • Valerie Quarmby
  • Ole L. Vesterqvist
  • David M. Potter
  • James L. Witliff
  • Herbert A. Fritche
  • Timothy O’Leary
  • Lorah Perlee
  • Sunil Kadam
  • John A. Wagner
Conference Report

DOI: 10.1007/s11095-005-2495-9

Cite this article as:
Lee, J., Weiner, R., Sailstad, J. et al. Pharm Res (2005) 22: 499. doi:10.1007/s11095-005-2495-9

No Heading

Biomarkers are increasingly used in drug development to aid scientific and clinical decisions regarding the progress of candidate and marketed therapeutics. Biomarkers can improve the understanding of diseases as well as therapeutic and off-target effects of drugs. Early implementation of biomarker strategies thus promises to reduce costs and time-to-market as drugs proceed through increasingly costly and complex clinical development programs. The 2003 American Association of Pharmaceutical Sciences/Clinical Ligand Assay Society Biomarkers Workshop (Salt Lake City, UT, USA, October 24–25, 2003) addressed key issues in biomarker research, with an emphasis on the validation and implementation of biochemical biomarker assays, covering from preclinical discovery of efficacy and toxicity biomarkers through clinical and postmarketing implementation. This summary report of the workshop focuses on the major issues discussed during presentations and open forums and noted consensus achieved among the participants on topics from nomenclature to best practices. For example, it was agreed that because reliable and accurate data provide the basis for sound decision making, biomarker assays must be validated in a manner that enables the creation of such data. The nature of biomarker measurements often precludes direct application of regulatory guidelines established for clinical diagnostics or drug bioanalysis, and future guidance on biomarker assay validation should therefore be adaptable enough that validation criteria do not stifle creative biomarker solutions.

Key Words:

biomarkersnonclinical and clinical drug developmentquantitative method development and validation

Abbreviations

AAPS

American Association of Pharmaceutical Sciences

BQL

below quantifiable limit

CDER

Center for Drug Evaluation and Research

CMS

Centers for Medicare & Medicaid Services

CLAS

Clinical Ligand Assay Society

CLIA

Clinical Lab Improvement Amendments

GLP

good laboratory practices

LBABFG

Ligand Binding Assay Bioanalytical Focus Group

LOD

lower limit of detection

LLOQ

lower limit of quantification

NCCLS

National Committee for Clinical Laboratory Standards

PD

pharmacodynamic

PK

pharmacokinetic

OIVD

Office of in Vitro Diagnostics Device Evaluation and Safety

QCs

quality controls

ULOQ

upper limit of quantification

Copyright information

© Springer Science+Business Media, Inc. 2005

Authors and Affiliations

  • Jean W. Lee
    • 1
  • Russ S. Weiner
    • 2
  • Jeff M. Sailstad
    • 3
  • Ronald R. Bowsher
    • 4
  • Dean W. Knuth
    • 5
  • Peter J. O’Brien
    • 6
  • Jean L. Fourcroy
    • 7
  • Rakesh Dixit
    • 8
  • Lini Pandite
    • 9
  • Robert G. Pietrusko
    • 10
  • Holly D. Soares
    • 11
  • Valerie Quarmby
    • 12
  • Ole L. Vesterqvist
    • 2
  • David M. Potter
    • 11
  • James L. Witliff
    • 13
  • Herbert A. Fritche
    • 14
  • Timothy O’Leary
    • 15
  • Lorah Perlee
    • 16
  • Sunil Kadam
    • 6
  • John A. Wagner
    • 8
  1. 1.MDS Pharma ServicesLincolnUSA
  2. 2.Bristol-Myers SquibbPrincetonUSA
  3. 3.Trimeris Inc.DurhamUSA
  4. 4.LINCO Diagnostic ServicesSt. CharlesUSA
  5. 5.Jasper ClinicKalamazooUSA
  6. 6.Eli Lilly and CompanyIndianapolisUSA
  7. 7.Walter Reed Army HospitalBethesdaUSA
  8. 8.Merck and Company, Inc.West PointUSA
  9. 9.GlaxoSmithKlineResearch Triangle ParkUSA
  10. 10.Millenium PharmaceuticalsCambridgeUSA
  11. 11.Pfizer Global ResearchGrotonUSA
  12. 12.Genetech, Inc.South San FranciscoUSA
  13. 13.University of LouisvilleLouisvilleUSA
  14. 14.MD Anderson HospitalUniversity of TexasAustinUSA
  15. 15.Food and Drug AdministrationRockvilleUSA
  16. 16.Molecular Staging Inc.New HavenUSA